Translate Bio Presents MRT5005 Data at the 33rd Annual North American Cystic Fibrosis Conference

NASHVILLE, Tenn., Oct. 31, 2019 (GLOBE NEWSWIRE) -- Translate Bio TBIO, a clinical-stage messenger RNA (mRNA) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, today presented data for MRT5005, an inhaled mRNA therapeutic, at the 33^rd Annual North American Cystic Fibrosis Conference (NACFC) taking place in Nashville, Tennessee from October 31 to November 2, 2019. MRT5005 is designed to address the underlying cause of cystic fibrosis (CF), regardless of genetic mutation by delivering mRNA encoding fully functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to cells in the lung through nebulization.

The poster presentation today and the oral presentation tomorrow describe the previously announced interim results from the single-ascending dose (SAD) portion of a first-in-human Phase 1/2 clinical trial of MRT5005 in 12 patients with CF. The multiple-ascending dose (MAD) portion of the clinical trial is ongoing with data expected in 2020.

"It's an exciting time in the field of CF treatment where significant advancements have been made for the majority of people with CF; however, there are still those with different mutations who do not benefit from these new medicines," said Jonathan Zuckerman, M.D., Director, Adult Cystic Fibrosis Program, Maine Medical Center and RESTORE-CF investigator. "These initial data are encouraging and highlight the potential for MRT5005 as a treatment for people with CF who currently have no treatment options."

The interim results describe data from 12 adult patients with CF who received a single dose of either MRT5005 or placebo (3:1 randomization). Patients who received MRT5005 were assigned to one of three dose groups (8, 16 or 24 mg). An overview of the results from the SAD portion of the study include:

• Marked increases in ppFEV₁ after a single MRT5005 dose were observed in 4 of 9 patients, primarily at the mid-dose level
• ppFEV₁ increases were observed in a patient considered non-amenable to current CFTR modulator regimens as well as in patients on CFTR modulator regimens
• Early improvement in ppFEV₁ after a single dose suggests MRT5005 is crossing the mucus layer and enabling production of functional CFTR protein
• No serious adverse events were reported at any dose level
• MRT5005 was generally well tolerated at low and mid-dose levels; primarily at high dose, patients receiving a single dose of MRT5005 experienced transient, mild to moderate febrile reactions
• Evaluation of immunogenicity markers showed no detection of anti-CFTR anti-drug antibodies or T-cell sensitization

A copy of the poster and oral presentation will be available on Translate Bio's investor website at the time of the data presentation.

About the Phase 1/2 Clinical Trial
The randomized, double-blind, placebo-controlled Phase 1/2 clinical trial of MRT5005 is designed to enroll up to 40 adult patients with CF who have two Class I and/or Class II mutations. The primary endpoint of the trial is to assess the safety and tolerability of single and multiple escalating doses of MRT5005 administered by nebulization. Percent predicted forced expiratory volume in one second (ppFEV₁), which is a well-defined and accepted endpoint measuring lung function, is also being measured at pre-defined timepoints throughout the trial. The Phase 1/2 clinical trial of MRT5005 for the treatment of CF is being conducted in collaboration with the Cystic Fibrosis Foundation Therapeutics Development Network.

About Translate Bio
Translate Bio is a clinical-stage mRNA therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction. The Company's MRT platform is designed to develop product candidates that deliver mRNA carrying instructions to produce intracellular, transmembrane and secreted proteins for therapeutic benefit. Translate Bio is primarily focused on applying its MRT platform to pulmonary diseases caused by insufficient protein production or where production of proteins can modify disease. The Company also believes its technology is applicable to a broad range of diseases, including diseases that affect the liver, eye and central nervous system. Additionally, the MRT platform may be applied to various classes of treatments, such as therapeutic antibodies or vaccines in areas such as infectious disease and oncology. Translate Bio's lead program is being developed as a treatment for cystic fibrosis (CF) and is in an ongoing Phase 1/2 clinical trial. For more information about the Company, please visit www.translate.bio or on Twitter at @TranslateBio.

Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include, but are not limited to, those regarding:  the potential for MRT5005 to address the underlying cause of CF; Translate Bio's expectation regarding the potential benefits and advantages of its technology; Translate Bio's plans to enroll patients in the Phase 1/2 clinical trial of MRT5005; and Translate Bio's plans to report data from the MAD portion of the Phase 1/2 clinical trial of MRT5005 in 2020. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "suggest," "target," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from current expectations and beliefs, including but not limited to: Translate Bio's ability to advance the development of its platform and programs under the timelines it projects, demonstrate the requisite safety and efficacy of its product candidates and replicate in later-stage clinical trials any positive findings from preclinical studies; the content and timing of decisions made by the FDA, other regulatory authorities and investigational review boards at clinical trial sites, including decisions as it relates to ongoing and planned clinical trials; Translate Bio's ability to obtain, maintain and enforce necessary patent and other intellectual property protection; the availability of significant cash required to fund operations; competitive factors; general economic and market conditions and other important risk factors set forth under the caption "Risk Factors" in Translate Bio's Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2019 filed with the Securities and Exchange Commission on July 31, 2019 and in any other subsequent filings made by Translate Bio. Any forward-looking statements contained in this press release speak only as of the date hereof, and Translate Bio specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.